Arrowhead Pharmaceuticals develops RNAi therapeutics that silence disease-causing genes, using its TRiM platform across cardiometabolic, cardiovascular, obesity and pulmonary diseases.
Arrowhead Pharmaceuticals, Inc. is a commercial-stage biopharmaceutical company (Nasdaq: ARWR) that develops medicines for intractable diseases by silencing the genes that cause them. Its approach rests on RNA interference (RNAi) therapeutics - a modality that acts at the genetic root of disease rather than at its symptoms. The company's technical work spans RNA chemistry, gene silencing, nucleic acid therapeutics, drug delivery and target gene knockdown.
At the centre of the science is the proprietary TRiM™ platform, which leverages a natural pathway to silence disease-causing genes. Combining a broad portfolio of RNA chemistries with efficient modes of delivery, TRiM is designed to trigger RNAi for rapid, deep and durable knockdown of target genes. Drug candidates built on the platform are advancing across a growing number of disease areas, among them cardiometabolic conditions, severe hypertriglyceridemia, cardiovascular disease, obesity and pulmonary diseases.
The clinical pipeline targets disease genes in the liver and lung, complemented by a pipeline of preclinical candidates. Part of the company's stated ambition is to take RNAi beyond the liver and to reach targets that small molecules and biologics cannot address - a distinction that opens the possibility of treating conditions in virtually any therapeutic area. Work of this kind suits scientists and clinicians intent on a defined problem: extending a precise therapeutic modality to diseases with a genetic basis that remain largely untreatable.
